Type I Diabetes Mellitus Gene Therapy

Type I diabetes mellitus (T1DM) affects more than 17 million people worldwide, and current treatments for T1DM, known as insulin therapy, require continued insulin injection, diet control, and constant monitoring of blood sugar. While gene therapy methods that restore insulin production in non-pancreatic cells might provide a one-shot cure for T1DB. We propose a gene therapy for T1DM using an adeno-associated viral (AAV) vector that transfects somatic cells with an insulin gene controlled by a glucose-sensitive promoter, thus potentially able to restore glucose-regulated insulin production in diabetic patients. Preliminary testing will be conducted on cell lines to assess the efficiency of the therapy.
With the aim of restoring insulin production in T1DM patients, we propose to genetically modify non-beta cells to produce insulin at high glucose concentration. An engineered AAV vector will deliver the insulin production system into 293 cell lines to be tested. The insulin gene has been cloned and modified to allow for efficient proteolytic cleavage in non-beta cells (furin). In initial tests mCherry will be used instead of insulin as a reporter, and the effect will be assessed using FACS and microscopy.

Click here to edit this page!

Project Description

Content

Type I diabetes mellitus (T1DM) affects more than 17 million people worldwide, and current treatments for T1DM, known as insulin therapy, require continued insulin injection, diet control, and constant monitoring of blood sugar. While gene therapy methods that restore insulin production in non-pancreatic cells might provide a one-shot cure for T1DB. We propose a gene therapy for T1DM using an adeno-associated viral (AAV) vector that transfects somatic cells with an insulin gene controlled by a glucose-sensitive promoter, thus potentially able to restore glucose-regulated insulin production in diabetic patients. Preliminary testing will be conducted on cell lines to assess the efficiency of the therapy.
With the aim of restoring insulin production in T1DM patients, we propose to genetically modify non-beta cells to produce insulin at high glucose concentration. An engineered AAV vector will deliver the insulin production system into 293 cell lines to be tested. The insulin gene has been cloned and modified to allow for efficient proteolytic cleavage in non-beta cells (furin). In initial tests mCherry will be used instead of insulin as a reporter, and the effect will be assessed using FACS and microscopy.

References

iGEM teams are encouraged to record references you use during the course of your research. They should be posted somewhere on your wiki so that judges and other visitors can see how you though about your project and what works inspired you.

You can use these subtopics to further explain your project

1. Overall project summary
2. Project Details
3. Materials and Methods
4. The Experiments
5. Results
6. Data analysis
7. Conclusions

It's important for teams to describe all the creativity that goes into an iGEM project, along with all the great ideas your team will come up with over the course of your work.

It's also important to clearly describe your achievements so that judges will know what you tried to do and where you succeeded. Please write your project page such that what you achieved is easy to distinguish from what you attempted.